Author Archives: Alice Melão

Tissue Remodeling, Toxic Molecule Accumulation Responsible for Upper Airway Involvement in MPS, Study Finds

Active tissue remodeling and treatment-resistant accumulation of toxic molecules are contributing factors in the development of persistent upper airway disease among patients with mucopolysaccharidosis (MPS), a study shows. The study, “Substrate accumulation and extracellular matrix remodelling promote persistent upper airway disease in mucopolysaccharidosis patients on enzyme…

Shire’s Investigational ERT Shows Limited Benefits for Sanfilippo Type A Children in Phase 2b Trial

Administration of an engineered version of the heparan-N-sulfatase (HNS) enzyme into the cerebrospinal fluid directly through the spinal canal failed to improve neurocognitive activity in infants with early-stage Sanfilippo syndrome type A, results from a Phase 2b trial show. However, the investigational enzyme replacement therapy (ERT) held some therapeutic activity…